Identification

Generic Name
AT2220
DrugBank Accession Number
DB05200
Background

AT2220 is an experimental, oral therapy for the treatment of Pompe disease and belongs to a class of molecules known as pharmacological chaperones. It is a small molecule designed to act as a pharmacological chaperone that specifically binds, stabilizes, and facilitates the proper folding and trafficking of α-glucosidase (GAA). GAA to the lysosome, where it can perform its normal function. AT2220 has been shown to increase GAA activity in cell lines derived from Pompe patients and in transfected cells expressing misfolded forms of GAA.

Type
Small Molecule
Groups
Investigational
Synonyms
Not Available

Pharmacology

Indication

Pompe disease, also known as glycogen storage disease type II or acid maltase deficiency, is a relatively rare neuromuscular and lysosomal storage disorder caused by inherited genetic mutations in a key enzyme called α-glucosidase (Gaa).

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Contraindications & Blackbox Warnings
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Pharmacodynamics

Not Available

Mechanism of action

AT2220 is designed to act as a pharmacological chaperone by selectively binding to the misfolded enzyme responsible for Pompe disease, Gaa. After binding to the enzyme, it is thought that AT2220 promotes the proper folding, processing, and trafficking of the enzyme from the endoplasmic reticulum to its final destination, the lysosome, the area of the cell where the enzyme does its work. Once it reaches the lysosome, the pharmacological chaperone is displaced, and the enzyme can perform its normal function, which is the breakdown of its natural substrate, glycogen.

TargetActionsOrganism
ULysosomal alpha-glucosidaseNot AvailableHumans
Absorption

Not Available

Volume of distribution

Not Available

Protein binding

Not Available

Metabolism
Not Available
Route of elimination

Not Available

Half-life

Not Available

Clearance

Not Available

Adverse Effects
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Toxicity

Not Available

Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Not Available
Food Interactions
Not Available

Categories

Drug Categories
Not Available
Classification
Not classified
Affected organisms
Not Available

Chemical Identifiers

UNII
0RN23C42QR
CAS number
Not Available
InChI Key
Not Available
InChI
Not Available
IUPAC Name
Not Available
SMILES
Not Available

References

General References
Not Available
PubChem Substance
347910020

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
2CompletedTreatmentPompe's Disease1
2TerminatedTreatmentPompe's Disease1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
Not Available
Prices
Not Available
Patents
Not Available

Properties

State
Solid
Experimental Properties
Not Available
Predicted Properties
Not Available
Predicted ADMET Features
Not Available

Spectra

Mass Spec (NIST)
Not Available
Spectra
Not Available

Targets

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Kind
Protein
Organism
Humans
Pharmacological action
Unknown
General Function
Maltose alpha-glucosidase activity
Specific Function
Essential for the degradation of glygogen to glucose in lysosomes.
Gene Name
GAA
Uniprot ID
P10253
Uniprot Name
Lysosomal alpha-glucosidase
Molecular Weight
105322.935 Da

Drug created at October 21, 2007 22:24 / Updated at June 12, 2020 16:52